Evolocumab (AMG145), inhibitor of an enzyme (PCSK9) that facilitates degradation of low-density lipoprotein (LDL) receptors, led to 60% greater reduction in LDL cholesterol than placebo in 331 patients with heterozygous familial hypercholesterolemia, reported a manufacturer-funded multicenter randomized trial (Raal FJ et al. Lancet.
doi:10.1016/S0140-6736(14)61399-4 [published online October 2, 2014]).
Familial hypercholesterolemia is thought to be the most common disorder inherited in autosomal-dominant fashion, affecting between 1 in 250 to 300 people worldwide and more than 3 million people in the United States and Europe. Intensive statin therapy often fails to reduce LDL cholesterol to levels sufficient to prevent premature cardiovascular disease for people with the disorder.
Anita Slomski. Evolocumab Lowers LDL in Genetic High Cholesterol Disorder. JAMA. 2014;312(20):2083. doi:10.1001/jama.2014.15519